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News: European Medicines Agency publishes first review of orphan designation

COMP, Last updated: 17/09/2010
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European Medicines Agency workshop on methodological aspects of clinical trials for efficacy evaluation in small populations

London, UK, 22/10/2002, Last updated: 12/11/2002
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COMP assesses whether Plenadren still meets orphan designation criteria: recommendation to maintain the period of market exclusivity at 10 years: Plenadren (hydrocortisone) for the treatment of adrenal insufficiency (PDF/113.5 KB) (updated)

Adopted
First published: 03/05/2016
Last updated: 25/11/2021
EMA/COMP/263073/2016
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Recommendation for maintenance of orphan designation at the time of marketing authorisation: Plenadren (hydrocortisone) for the treatment of adrenal insufficiency (PDF/112.18 KB) (updated)

Adopted
First published: 15/11/2011
Last updated: 25/11/2021
EMA/729720/2011
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EU/3/06/372: Public summary of positive opinion for orphan designation of hydrocortisone (modified release tablet) for the treatment of adrenal insufficiency (PDF/194.75 KB) (updated)

Adopted
First published: 21/09/2009
Last updated: 25/11/2021
EMEA/COMP/137594/2006 Rev.4
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Recommendation for maintenance of orphan designation at the time of marketing authorisation: Vyndaqel (N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate) for treatment ... (PDF/113.47 KB) (updated)

Adopted
First published: 21/12/2011
Last updated: 25/11/2021
EMA/COMP/729178/2011
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EU/3/06/401: Public summary of positive opinion for orphan designation of N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate for the treatment of familial amyloid polyne... (PDF/195.36 KB) (updated)

Adopted
First published: 02/04/2009
Last updated: 25/11/2021
EMEA/COMP/186264/2008 Rev. 3
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EU/3/15/1541: Public summary of opinion on orphan designation: Ibrutinib for the treatment of marginal zone lymphoma (PDF/184.17 KB) (updated)

Adopted
First published: 02/10/2015
Last updated: 23/11/2021
EMA/COMP/508544/2015
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EU/3/13/1203: Public summary of opinion on orphan designation: Ibrutinib for the treatment of diffuse large B-cell lymphoma (PDF/185.93 KB) (updated)

Adopted
First published: 02/12/2013
Last updated: 23/11/2021
EMA/COMP/629119/2013
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EU/3/13/1212: Public summary of opinion on orphan designation: Ibrutinib for the treatment of follicular lymphoma (PDF/180.21 KB) (updated)

Adopted
First published: 23/01/2014
Last updated: 23/11/2021
EMA/COMP/708125/2013
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EU/3/12/987: Public summary of opinion on orphan designation: (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt for the treatment of myelodysplastic syndromes (PDF/200.73 KB)

Adopted
First published: 18/06/2012
Last updated: 27/10/2021
EMA/COMP/217857/2012 Rev. 1
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EU/3/18/2038: Public summary of opinion on orphan designation: Palovarotene for the treatment of multiple osteochondromas (PDF/180.32 KB)

First published: 21/08/2018
Last updated: 19/10/2021
EMA/371472/2018
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EU/3/05/333: Public summary of positive opinion for orphan designation of eptacog alpha (activated) for the treatment of diffuse alveolar haemorrhage (PDF/194.94 KB)

Adopted
First published: 01/01/2006
Last updated: 21/09/2021
EMEA/COMP/370803/2005 Rev. 5
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EU/3/08/565: Public summary of positive opinion for orphan designation of drotrecogin alfa (activated) for the treatment of acute respiratory distress syndrome (PDF/204.45 KB)

Adopted
First published: 24/02/2009
Last updated: 21/09/2021
EMEA/COMP/441380/2008 Rev. 3
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EU/3/13/1214: Public summary of opinion on orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabi... (PDF/214.73 KB)

Adopted
First published: 22/01/2014
Last updated: 08/09/2021
EMA/COMP/696783/2013 Corr.
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EU/3/13/1154: Public summary of opinion on orphan designation: Fosbretabulin tromethamine for the treatment of ovarian cancer (PDF/211.9 KB)

Adopted
First published: 21/08/2013
Last updated: 02/09/2021
EMA/COMP/417559/2013
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EU/3/15/1560: Public summary of opinion on orphan designation: N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide for the treatment of Duchenne muscular dystrophy (PDF/216.55 KB)

Adopted
First published: 18/11/2015
Last updated: 02/09/2021
EMA/COMP/603544/2015
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EU/3/15/1487: Public summary of opinion on orphan designation: Reduced oxidised N-acetyl heparin for the treatment of plasma cell myeloma (PDF/196.74 KB)

Adopted
First published: 30/06/2015
Last updated: 02/09/2021
EMA/COMP/281341/2015
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EU/3/02/094: Public summary of positive opinion for orphan designation of chimeric IgG monoclonal antibody cG250 for the treatment of renal cell carcinoma (PDF/259.87 KB)

Adopted
First published: 01/08/2003
Last updated: 02/09/2021
EMEA/COMP/76/02 Rev. 1
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EU/3/08/582: Public summary of positive opinion for orphan designation of recombinant human heparan-N-sulfatase for the treatment of mucopolysaccharidosis III, type A (Sanfilippo A syndrome) (PDF/317.72 KB)

Adopted
First published: 29/06/2009
Last updated: 02/09/2021
EMEA/COMP/488980/2008
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Lumoxiti : Orphan maintenance assessment report (PDF/922.17 KB)

Adopted
First published: 09/03/2021
Last updated: 11/08/2021
EMADOC-1700519818-608352
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EU/3/04/227: Public summary of positive opinion for orphan designation of l, 1'-[1,4-phenylenebis (methylene)]-bis-1,4,8,11- tetraazacyclotetradecane for treatment to mobilize progenitor cells prior to stem cell tran... (PDF/305.31 KB)

Adopted
First published: 18/08/2008
Last updated: 10/08/2021
EMEA/COMP/77477/2004 Rev.1
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EU/3/04/195: Public summary of positive opinion for orphan designation of 2-methoxy-5-[(1Z)-2-(3,4,5-trimethoxyphenyl) ethenyl]-phenol for the treatment of anaplastic thyroid cancer (PDF/196.2 KB)

Adopted
First published: 05/11/2004
Last updated: 21/07/2021
EMEA/COMP/136/04 rev. 1
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EU/3/15/1434: Public summary of opinion on orphan designation: Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 regions for the treatment of ovarian cancer (PDF/199.18 KB)

Adopted
First published: 08/04/2015
Last updated: 25/06/2021
EMA/COMP/49562/2015
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EU/3/14/1410: Public summary of opinion on orphan designation: Recombinant human aspartylglucosaminidase for the treatment of aspartylglucosaminuria (PDF/190.99 KB)

Adopted
First published: 05/03/2015
Last updated: 25/06/2021
EMA/COMP/10141/2015

2247 results

News: European Medicines Agency publishes first review of orphan designation

European Medicines Agency workshop on methodological aspects of clinical trials for efficacy evaluation in small populations

COMP assesses whether Plenadren still meets orphan designation criteria: recommendation to maintain the period of market exclusivity at 10 years: Plenadren (hydrocortisone) for the treatment of adrenal insufficiency (PDF/113.5 KB) (updated)

Recommendation for maintenance of orphan designation at the time of marketing authorisation: Plenadren (hydrocortisone) for the treatment of adrenal insufficiency (PDF/112.18 KB) (updated)

EU/3/06/372: Public summary of positive opinion for orphan designation of hydrocortisone (modified release tablet) for the treatment of adrenal insufficiency (PDF/194.75 KB) (updated)

Recommendation for maintenance of orphan designation at the time of marketing authorisation: Vyndaqel (N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate) for treatment ... (PDF/113.47 KB) (updated)

EU/3/06/401: Public summary of positive opinion for orphan designation of N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate for the treatment of familial amyloid polyne... (PDF/195.36 KB) (updated)

EU/3/15/1541: Public summary of opinion on orphan designation: Ibrutinib for the treatment of marginal zone lymphoma (PDF/184.17 KB) (updated)

EU/3/13/1203: Public summary of opinion on orphan designation: Ibrutinib for the treatment of diffuse large B-cell lymphoma (PDF/185.93 KB) (updated)

EU/3/13/1212: Public summary of opinion on orphan designation: Ibrutinib for the treatment of follicular lymphoma (PDF/180.21 KB) (updated)

EU/3/12/987: Public summary of opinion on orphan designation: (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt for the treatment of myelodysplastic syndromes (PDF/200.73 KB)

EU/3/18/2038: Public summary of opinion on orphan designation: Palovarotene for the treatment of multiple osteochondromas (PDF/180.32 KB)

EU/3/05/333: Public summary of positive opinion for orphan designation of eptacog alpha (activated) for the treatment of diffuse alveolar haemorrhage (PDF/194.94 KB)

EU/3/08/565: Public summary of positive opinion for orphan designation of drotrecogin alfa (activated) for the treatment of acute respiratory distress syndrome (PDF/204.45 KB)

EU/3/13/1214: Public summary of opinion on orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabi... (PDF/214.73 KB)

EU/3/13/1154: Public summary of opinion on orphan designation: Fosbretabulin tromethamine for the treatment of ovarian cancer (PDF/211.9 KB)

EU/3/15/1560: Public summary of opinion on orphan designation: N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide for the treatment of Duchenne muscular dystrophy (PDF/216.55 KB)

EU/3/15/1487: Public summary of opinion on orphan designation: Reduced oxidised N-acetyl heparin for the treatment of plasma cell myeloma (PDF/196.74 KB)

EU/3/02/094: Public summary of positive opinion for orphan designation of chimeric IgG monoclonal antibody cG250 for the treatment of renal cell carcinoma (PDF/259.87 KB)

EU/3/08/582: Public summary of positive opinion for orphan designation of recombinant human heparan-N-sulfatase for the treatment of mucopolysaccharidosis III, type A (Sanfilippo A syndrome) (PDF/317.72 KB)

Lumoxiti : Orphan maintenance assessment report (PDF/922.17 KB)

EU/3/04/227: Public summary of positive opinion for orphan designation of l, 1'-[1,4-phenylenebis (methylene)]-bis-1,4,8,11- tetraazacyclotetradecane for treatment to mobilize progenitor cells prior to stem cell tran... (PDF/305.31 KB)

EU/3/04/195: Public summary of positive opinion for orphan designation of 2-methoxy-5-[(1Z)-2-(3,4,5-trimethoxyphenyl) ethenyl]-phenol for the treatment of anaplastic thyroid cancer (PDF/196.2 KB)

EU/3/15/1434: Public summary of opinion on orphan designation: Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 regions for the treatment of ovarian cancer (PDF/199.18 KB)

EU/3/14/1410: Public summary of opinion on orphan designation: Recombinant human aspartylglucosaminidase for the treatment of aspartylglucosaminuria (PDF/190.99 KB)